A new hope for epidermolysis bullosa (“butterfly children”) thanks to novel CRISPR-Cas3 platform
Osaka, Japan, and Lugano, Switzerland, October 30th, 2023 – C4U Corporation (C4U) and Healiva SA(Healiva) are pleased to announce that they have entered into a strategic alliance agreement regarding
the development of CRISPR-Cas3 derived therapeutic solutions for Epidermolysis Bullosa (EB), also known
as “butterfly children”.
Epidermolysis bullosa is a rare genetic skin disorder characterized by highly sensitive skin that blisters anderodes with minimal friction or trauma, such as rubbing or scratching. This condition predominantly
affects young children, causing them to experience severe pain and a substantial reduction in their qualityof life due to the constant formation of blisters in response to minor injuries. EB affects approximately 20
out of every 1 million newborns in the United States where 25,000 to 50,000 young children are livingwith this debilitating condition.
“The collaboration aims to provide answers and hope to the individuals affected by EB, a devastating skincondition that renders the skin of the affected children as fragile as butterfly wings,” said Valerio M. Ferrari,
Bioseutica Group Chairman & Co-Founder of Healiva.
The collaborative endeavor between Healiva and C4U will focus on the development of CRISPR-Cas3 based
gene therapy for EB. In this collaboration the parties will work together to develop a gene therapy for EB
with a staged development process to maximize speed and approval to perform clinical studies. By
combining their complementary expertise, resources, and shared vision, both entities will strategicallycollaborate to enhance the quality of life for young children globally affected worldwide by this rare skincondition.
“By utilizing its proprietary CRISPR-Cas3 technology, a new generation of gene editing technology, C4U’sfocus is to develop alternative and complete cures to genetic diseases. Skin diseases, especially EB, arerelevant targets for the CRISPR-Cas3 platform, and we believe that we will bring a bright future to EB
patients throughout the world,” said Akimitsu Hirai, President & CEO of C4U.
"This collaboration integrates gene therapy prospects with our comprehensive cell therapy portfolio,
leveraging our expertise in autologous (Epidex®) and allogenic therapy technologies and the capabilitiesof our manufacturing infrastructure," articulated Dr. Priyanka Dutta Passecker, Co-Founder & CEO ofHealiva.
About Healiva SA
Healiva SA (www.healiva.com) is a Lugano, Switzerland-based biotechnology company founded by
Bioseutica and Dr. Priyanka Dutta Passecker in 2020.
Healiva’s Wound Care & Skin Diseases proprietary technology platform, including all related
manufacturing technologies and automation processes, is focused on therapies and cures for acute andchronic wounds. The affordable end-to-end approach by combining cell therapies, autologous (EpiDex®:
an autologous epidermis derived from the patient’s own hair cells) and allogenic cell therapy (an optimal
mixture of skin fibroblasts and keratinocytes produce and release active growth factors into the wound,stimulating the patient’s own cells to repair and close the wound) and medical devices to deliver
personalized and precision medicine in wound care.
About C4U Corporation
C4U Corporation (www.crispr4u.jp/en/) is a privately held biotech company based in Osaka, Japan, andfocused on the development of safe and efficient gene therapies for rare diseases, including pediatric,
based on its proprietary next generation CRISPR-Cas3 gene editing platform. The CRISPR-Cas3 technology
platform is similar to CRISPR-Cas9 in terms of its engineering, efficiency, and delivery methods and has
already been validated both in vitro and in vivo. It presents the distinct benefits of: (1) no off-target
deletions (improved safety); (2) efficient knockouts of large, programmable gene sequences; and (3)entirely independent patent portfolio, which has been exclusively licensed worldwide to C4U by Osaka
University for use in eukaryotic cells, thus simplifying sublicensing transactions in sharp contrast with thecomplex and heavily litigated CRISPR-Cas9 patent landscape.
Media Contacts:
C4U Corporation: Katsuyoshi Akiyama, PhD, Director of Business Development (info@crispr4u.com)
Healiva SA: Priyanka Dutta Passecker, PhD, CEO (contact@healiva.com)